What the EU pharmaceutical reform means for children’s medicines
Brussels’ overhaul of its medicines legislation folds the Paediatric Regulation into a single framework. The details will decide whether plans for children start earlier.
In brief
The EU pharma reform folds the Paediatric Regulation into one framework. What it means for paediatric investigation plans and mechanism-of-action rules.
- The Commission proposed the package in April 2023, replacing much of the EU’s medicines law.
- Paediatric and orphan rules are being folded into the general legislation.
- A shift towards mechanism-of-action-based paediatric plans is one of the most consequential changes for developers.
In April 2023 the European Commission proposed the biggest rewrite of EU pharmaceutical law in two decades: a new directive and a new regulation covering everything from data protection periods to shortages. Folded into it are the rules that have governed children’s medicines since 2007.
The package has since worked its way through the EU’s legislative process. The European Parliament adopted its position in 2024 and member states agreed theirs in 2025, opening negotiations on a final text.
Why paediatrics was in scope
The Commission’s own evaluation of the Paediatric and Orphan Regulations, published in 2020, found that the paediatric rules had increased the number of medicines with information for children. It also found that they had done little to drive development in areas where children’s needs differ from adults’, and that the process for agreeing paediatric investigation plans had become slow and heavy.
The changes that matter most
The proposals aim to make paediatric investigation plans more flexible and quicker to agree, with a simplified procedure for some products and more room to adapt plans as evidence develops. Deferrals are meant to be better justified and better tracked.
The most consequential change for developers is the move towards requiring paediatric development based on a medicine’s mechanism of action. Under the current rules, a waiver can be granted when the adult condition does not occur in children. That has allowed drugs whose biology could matter for a different paediatric disease to skip children entirely. The US closed a similar loophole for oncology with the RACE for Children Act.
What to watch
For companies, the practical questions are timing and predictability. Will plans need to be agreed earlier? Will the mechanism-of-action test be applied narrowly to oncology, or more broadly? And how will rewards for completed paediatric work interact with the new, modulated periods of regulatory data protection?
The answers will shape development plans for products entering the clinic now, which will be the first to run their paediatric programmes under the new rules.
